for people ages 18-65 (full criteria)
at Sacramento, California and other locations
study started
completion around
Principal Investigator
by Valentina Medici, Dr



The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult patients with Wilson's Disease prior to and following background WD therapy withdrawal.

Official Title

A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients With Wilson's Disease


Wilson's Disease, Hepatolenticular Degeneration, VTX-801


You can join if…

Open to people ages 18-65

  • Male or female aged 18 and 65 years inclusive
  • Confirmed diagnosis of WD
  • Treated for WD according to international recommendations with no current evidence for inadequate treatment
  • Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder and (ii) Stable laboratory parameters used to assess copper metabolism

You CAN'T join if...

  • ALT level ≥ 2 ULN that is not readily explained by extrinsic factors
  • Total bilirubin > 1.5 x ULN in the absence of proven Gilbert's syndrome; in case of Gilbert's syndrome, direct bilirubin > ULN
  • INR > 1.2
  • Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrollment visit
  • Patient has moderate or severe renal impairment defined as eGFR CKD-EPI < 60 mL/min/1.73 m2, or patient has nephritis or nephrotic syndrome
  • Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection
  • Any history or current evidence of hepatitis B infection
  • Any history of hepatitis C infection, unless previous viral RNA assays in two samples, collected at least 6 months apart, are negative
  • Positive QuantiFERON®-TB Gold tuberculosis test result
  • Any concomitant disorder/condition - including hepatic disorders - or treatment possibly interfering with the conduct or evaluation of the study
  • Any history of diabetes
  • Pregnancy or breastfeeding
  • Body Mass Index ≥ 35 kg/m2

    Other protocol defined Inclusion/ Exclusion criteria may apply


  • UC Davis Medical Center accepting new patients
    Sacramento California 95817 United States
  • University of Texas Southwestern Medical Center accepting new patients
    Dallas Texas 75235 United States

Lead Scientist at UC Davis

  • Valentina Medici, Dr
    Professor, Gastroenterology and Hepatology, School of Medicine. Authored (or co-authored) 129 research publications


accepting new patients
Start Date
Completion Date
Vivet Therapeutics SAS
Sign up for this study
Phase 1/2 Wilson's Disease Research Study
Study Type
Expecting 16 study participants
Last Updated